Splicing Modulation as a Promising Therapeutic Strategy for Lysosomal Storage Disorders: The Mucopolysaccharidoses Example

Keyphrases

• Therapeutic Strategies 100%
• Lysosomal Storage Disease 100%
• Photosystem II 100%
• Splicing Modulation 100%
• Aberrant Splicing 40%
• Enzyme Activity 20%
• Gene Expression 20%
• MRNA Level 20%
• Therapeutic Potential 20%
• RNA Splicing 20%
• Drug Use 20%
• New Therapeutic Strategies 20%
• Monogenic 20%
• Genetic Disease 20%
• Specific Populations 20%
• Pathogenic Variants 20%
• General Mechanism 20%
• Nucleoside Analogues 20%
• Pre-mRNA 20%
• RNA Species 20%
• Splicing Process 20%
• Splicing Pattern 20%
• Splicing Defect 20%
• Modulation Approach 20%
• SiRNA Drugs 20%
• RNA Biology 20%
• Complementary Base Pairing 20%
• Marketed Formulations 20%

Pharmacology, Toxicology and Pharmaceutical Science

• Storage Disease 100%
• Lysosome Storage Disease 100%
• Mucopolysaccharidosis 100%
• Disease 40%
• Messenger RNA Precursor 40%
• Base 20%
• Nucleic Acid 20%
• Genetic Disorder 20%
• Preclinical Study 20%
• Monogenic Disorder 20%

Biochemistry, Genetics and Molecular Biology

• RNA 100%
• Mucopolysaccharidosis 100%
• Lysosomal Storage Disease 66%
• Genetics 16%
• Enzyme Activity 16%
• RNA Splicing 16%
• Gene Expression 16%
• Genetic Disorder 16%
• Preclinical Study 16%
• Base Pairing 16%
• Precursor mRNA 16%
• Splicing Defect 16%
• Nucleic Acid Analogue 16%